Feb. 15 is International Angelman Syndrome Day, and the Synk family aims to raise awareness and honor people with the rare ...

For the last several months, and throughout the majority of the NFL season, the Kansas City skyline has illuminated a deep cardinal red and golden hue in honor of the Chiefs. However, things will ...

Feb. 15 is International Angelman Syndrome Day, and the Synk family aims to raise awareness and honor people with the rare ...

Rare diseases have become an attractive target for pharmaceutical companies both in Australia and internationally with ...

Total Revenue of $560 million, exceeding guidanceCrysvita® revenue of $410 million and Dojolvi® revenue of $88 million2025 Financial ...

Emil Kakkis; President, Chief Executive Officer, Director; Ultragenyx Pharmaceutical Inc Erik Harris; Executive Vice President, Chief Commercial Officer; Ultragenyx Pharmaceutical Inc Howard Horn; ...

Reports Q4 revenue $164.88M, consensus $158.71M. “We have created a next-generation rare disease company on a pathway to profitability with ...

Encoded Therapeutics is shrinking its headcount by 29% as the gene therapy biotech draws resources away from its early-stage ...

D. Boral Capital reaffirmed their buy rating on shares of Anavex Life Sciences (NASDAQ:AVXL – Free Report) in a report published on Wednesday morning,Benzinga reports. D. Boral Capital currently has a ...

Encoded’s layoffs will mostly affect its technology and early-stage research and development functions. The move is expected ...

Ionis has committed to a phase 3 programme for its antisense therapy for rare genetic disorder Angelman syndrome, just weeks after Biogen decided against exercising an option to license the drug.